Review Article: Rome Foundation Working Team Report: Consensus Statement on the Design and Conduct of Behavioural Clinical Trials for Disorders of Gut–Brain Interaction

Background: Brain–gut behaviour therapies (BGBT) have gained widespread acceptance as therapeutic modalities for the management of disorders of gut–brain interaction (DGBI). However, existing treatment evaluation methods in the medical field fail to capture the specific elements of scientific rigour unique to behavioural trial evaluation. Aims: To offer the first consensus on the development and testing of BGBT in DGBI. Methods: An international, interdisciplinary team of experts developed a consensus statement heavily informed by best practice recommendations for behavioural clinical trials for chronic diseases, organised by a selected treatment development model. Results: We suggest an existing behavioural treatment development model that has an iterative progression aligned with the drug development model with nuances specific to BGBT. We describe the iterative phases through initial discovery and experimental work, assembly of a mechanistic pathway and candidate treatment components, treatment refinement and optimisation, initial proof-of-concept, feasibility of clinical trials and, finally, confirmatory efficacy and effectiveness testing. We delineate recommendations for and provide examples that lend themselves to gastroenterologists planning to develop or test BGBT, reviewing proposals for or results from BGBT studies or writing management guidelines for DGBI. Conclusions: This working team report facilitates a shared understanding of the elements of scientific rigour necessary for BGBT development and could support future standards on which BGBT are evaluated in gastroenterology.